"Excellent graphic for the 4 ways to suppress gene expression http://www.nejm.org/doi/full/10.1056/NEJMcibr1716741 … by @AmmarAlChalabi @NEJM with specific attention to CRISPR genome editing for ALS" Eric Topol
Finding a Treatment for ALS — Will Gene Editing Cut It? Ammar Al-Chalabi, F.R.C.P., Ph.D., and Robert H. Brown, M.D., D.Phil. N Engl J Med 2018; 378:1454-1456
AI Hunts for New ALS Treatments Scientific American By Ben Hirschler, Reuters on August 10, 2017
"Artificially intelligent machines could analyze huge databases to find potential targets for the devastating disease"
"One candidate proposed by AI machines recently produced promising results in preventing the death of motor neurone cells and delaying disease onset in preclinical tests in Sheffield."
ENCALS (European Network to Cure ALS) meeting 2017 Ljubljana, Slovenia 20 May 2017
AB Science trumpets phase 3 ALS data, but investors seem unmoved FierceBiotech May 19, 2017
New gene variant identified in ALS provides treatment hope Drug Target Review 17 May 2017
"A new study shows that a variant in UBQLN4 gene has been associated with Lou Gehrig’s disease, or amyotrophic lateral sclerosis (ALS). The study also describes how this gene variant disrupts a cellular process that drives motor neuron development. This new insight opens the door to potential treatment targets for ALS.
BrainStorm Initiated Technology Transfer to City of Hope for U.S. Production of NurOwn® for Phase 3 ALS Study BrainStorm Cell Therapeutics May 16, 2017
AB Science Seeks to Shed Skepticism With ALS Drug Data Reveal The Street May 9, 2017
Mitsubishi's Radicava, first ALS med in decades, beats all comers to FDA approval FiercePharma May 8, 2017
"The FDA was so impressed with Mitsubishi Tanabe's amyotrophic lateral sclerosis drug, it recruited the company to file its data for U.S. approval based solely on data generated in Japan. Now, with a Friday approval for Radicava, ALS patients in the U.S. will have their first new treatment option in 22 years—at the prediscount price of $145,000 per year."
FDA approves first ALS treatment in more than two decades Reuters Fri May 5, 2017
"The Food and Drug Administration on Friday approved Mitsubishi Tanabe Pharma Corp's treatment for fatal neurological disorder amyotrophic lateral sclerosis (ALS), marking the first such U.S. regulatory approval in more than two decades.
The drug, known chemically as edaravone, has been sold by Japan-based Mitsubishi Tanabe in Japan and South Korea since 2015."
Brainstorm Reports on Successful End of Phase 2 Meeting with FDA, Upcoming Phase 3 Trial and Planned Application for Hospital Exemption for NurOwn® BrainStorm Cell Therapeutics Dec. 19, 2016
MediciNova annonce résultats exploratoires intérimaires et cliniques de l’essai clinique de MN-166 (ibudilast) en SLA présentés lors du 27e Symposium International ALS/MND à Dublin, Irlande ALS 15-12-2016
EC Grants Orphan Medicinal Product Designation for MN-166 (ibudilast) for Amyotrophic Lateral Sclerosis CheckOrphan December 22, 2016
Yuichi Iwaki, MD, PhD, President and Chief Executive Officer of MediciNova, Inc., commented, "We are very pleased to receive orphan medicinal product designation for MN-166 for ALS in the European Union which complements our recently granted orphan-drug designation for ALS in the U.S. Interim data from the mid-study analysis of our ongoing clinical trial of MN-166 in ALS at Carolinas HealthCare System's Neuromuscular/ALS-MDA Center, along with previously reported positive ALS preclinical study data, was submitted to EMA COMP to address the scientific rationale for orphan medicinal product designation to establish the medical basis for the use of MN-166 (ibudilast) for ALS."
12/09/16 BrainStorm Cell Therapeutics - Session: Late Breaking News - PDF: US clinical trial BCT-001-US
Title: A Phase 2, Randomized, Double Blind, Placebo Controlled Multicenter Study to Evaluate Safety and Efficacy of Transplantation of Autologous Mesenchymal Stem Cells Secreting Neurotrophic Factors in Patients with ALS
Matthew Disney Receives New Grant to Investigate Myotonic Dystrophy and ALS The Scipps Research Institute Dec 2016
For more information on this research, see Matthew Disney’s Biosketch and the Disney Laboratory Website.
(v) Developing specific lead therapeutics that reduce the production of toxic proteins that are known to cause the majority of cases of Amyotrophic Lateral Sclerosis (ALS, Lou Gehrig’s disease) and Frontotemporal Dementia
"BrainStorm Cell Therapeutics Inc. (BCLI), a leading developer of adult stem cell technologies for neurodegenerative diseases, announced today data from the Company's Phase 2 study of NurOwn® in ALS, will be highlighted in presentations at the 27th International Symposium on ALS/MND, being held December 7-9, 2016 in Dublin, Ireland."
Tuesday, December 6 - Are Stem Cells Ready for Human Clinical Trials? Anthony J. Windebank, MD, Professor of Neurology, The Mayo Clinic, and Principal Investigator of the Phase 2 Trial of NurOwn
Friday, December 9 - A Phase 2, Randomized, Double Blind, Placebo Controlled Multicenter Study to Evaluate Safety and Efficacy of Transplantation of Autologous Mesenchymal Stem Cells Secreting Neurotrophic Factors in Patients with Amyotrophic Lateral Sclerosis. James D. Berry, MD, MPH, Department of Neurology, Massachusetts General Hospital, and Principal Investigator of the Phase 2 NurOwn Trial
Stephen Hawking hospitalised in Rome for checks Reuters Dec 2, 2016
"The brain–computer interface offered autonomous communication that supplemented and at times supplanted the patient’s eye-tracking device."
ALS Clinical Trials - Essais Thérapeutiques sur la SLA FISLAN (Actualisation octobre 2016).
"I really wanted to attend this conference" Bryan Hankey 2015 State of ALS Research